Ibrutinib Granted Supplemental New Drug Application for Chronic GVHD

The U.S. Food and Drug Administration (FDA) accepted a supplemental new drug application of ibrutinib for patients with chronic graft-versus-host disease (cGVHD) who have not responded to ≥1 lines of systemic therapy.

The decision was based on data from the single arm, phase Ib/II PCYC-1129 trial, which was presented at the 2016 ASH Annual Meeting. In a cohort of 42 previously treated patients with cGVHD, ibrutinib was associated with an overall response rate of 67 percent and showed clinically meaningful and durable responses. Twenty-one percent of responders had a complete response (CR) and 45 percent had a partial response.

Patients were included in the study if they had not responded to ≤3 prior therapies for cGVHD and had either an erythematous rash on >25 percent of their body surface area or a National Institutes of Health Oral Mucosal Score of >4.

Patients received oral ibrutinib 420 mg daily in combination with ongoing therapies, including corticosteroids and other immunosuppressants.

Most patients (71%) had a sustained cGVHD response of at least 5 months. cGVHD response was observed across multiple organs: 56 percent (n=20/25) of patients with ≥2 involved organs at baseline responded in at least two organs, and 42 percent of patients with ≥3 involved organs at baseline responded in at least three organs.

In June 2016, the FDA granted ibrutinib breakthrough-therapy and orphan drug designation as a potential treatment for cGVHD after failure of ≥1 lines of systemic therapy.

Source: Janssen Research & Development, LLC, news release, April 5, 2017.